Immunotherapy with so-called CAR-T cells has established itself for the treatment of various leukemias (blood cancer) and lymphomas (lymph gland cancer). A team led by Dr. Kai Rejeski and Prof. Marion Subklewe from the Medical Clinic III of the LMU Klinikum found that many patients suffer from a side effect that initially fell under the radar: a
Blood Cancer Discovery, a journal of the American Association for Cancer Research, has published a research article demonstrating a novel approach that may reduce a serious adverse effect associated with chimeric antigen receptor (CAR) T-cell therapy, a form of immunotherapy. This research was presented at the American Society of Hematology Annual Meeting in Atlanta, Georgia
Scientists at Boston Children’s Hospital have successfully used gene therapy to reverse a form of genetic hearing loss in mice. The team corrected a gene mutation that affects sensory hair cells in the inner ear, and the new jigsaw-like method could help improve gene therapy for other disorders. The target of the new treatment was
Mount Sinai scientists have become the first to report a potentially serious side effect related to a new form of immunotherapy known as CAR-T cell therapy, which was recently approved for the treatment of multiple myeloma. Their findings were published as a case study in Nature Medicine in December. Multiple myeloma is a complex and incurable type
Broad and robust transgene expression across brain regions of marmoset is shown after systemic delivery with engineered capsid. Gene therapy is a powerful developing technology that has the potential to address myriad diseases. For example, Huntington’s disease, a neurodegenerative disorder, is caused by a mutation in a single gene, and if researchers could go into
Clinical trial planning underway to test the widely used diabetes drug metformin as a preventive treatment for the blinding eye disease Scientists have discovered that gene therapy and the diabetes drug metformin may be potential treatments for late-onset retinal degeneration (L-ORD), a rare, blinding eye disease. Researchers from the National Eye Institute (NEI), part of
The CompacT SelecT (CTST) platform is a modular robotic system that integrates a full range of cell culture proceduresunder sterile conditions that mimic the manual cell culture process. These procedures include automated handling of different cell culture vessels, pipetting large and small volumes at adjustable speeds, cell counting, cell viability analysis, cell density assessment, microscopic
Rapid advances in the development of cell and gene therapies over the past several years have heralded “an evolutionary time in medicine.” That’s the opinion of Peter Walters, Director of Advanced Therapies at CRB. That thought reflects data seen in CRB’s annual Horizon Life Sciences report that highlights the rapid expansion of the development of cell and
About 20,000 people in the U.S. live with hemophilia A. It’s a rare X-linked genetic disorder that affects predominantly males and causes their blood to clot poorly when healing wounds. For some, routine daily activities can turn into painful medical emergencies to stop internal bleeding, all because of changes in a single gene that disables
The first tests on eight children show favorable preliminary results, with an adjustment of motor and cognitive skills and improvements in some brain and joint parameters Gene therapy takes steps forward in the fight against rare diseases. Eight children with severe Hurler’s syndrome, a disease associated with reduced life expectancy, benefited from this therapy. The
